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Formulation and PEGylation optimization of the therapeutic PEGylated phenylalanine ammonia lyase for the treatment of phenylketonuria

Phenylketonuria (PKU) is a genetic metabolic disease in which the decrease or loss of phenylalanine hydroxylase (PAH) activity results in elevated, neurotoxic levels of phenylalanine (Phe). Due to many obstacles, PAH enzyme replacement therapy is not currently an option. Treatment of PKU with an alt...

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Detalles Bibliográficos
Autores principales: Bell, Sean M., Wendt, Dan J., Zhang, Yanhong, Taylor, Timothy W., Long, Shinong, Tsuruda, Laurie, Zhao, Bin, Laipis, Phillip, Fitzpatrick, Paul A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5345807/
https://www.ncbi.nlm.nih.gov/pubmed/28282402
http://dx.doi.org/10.1371/journal.pone.0173269